Ocular Melanoma Foundation

Latest New Medical Therapy Trial Results

Following are the most recent results for new drug therapies currently in clinical trials worldwide. Results are also searchable by therapeutic area beginning with the most recent updates.

Week of September 18, 2017

Advicenne issued results of a phase III trial of ADV7103 in adults and children suffering from distal Renal Tubular Acidosis (dRTA). ADV7103 is given twice a day in contrast to the current standard of care (SoC), which are usually various unapproved products administered every four to six hours to attempt to re-balance the body’s pH and to normalize blood potassium level (kalaemia). The phase III study of ADV7103 was shown to restore the main biological defects observed, with the disease meeting positively primary and secondary endpoints. The objective of the study was to evaluate the efficacy, safety and acceptability of ADV7103, an innovative oral product, formulated in pediatric-friendly coated granules, that combines two active pharmaceutical ingredients. Normal blood bicarbonate levels were attained in most patients treated with doses of ADV7103 ranging from 0.75 to 8.45 mEq/kg/day. Mean doses of 1.7, 2.3, 3.8 and 6.1 mEq/kg/day ADV7103 were given, respectively, in adults, adolescents, children and infants. Non-inferiority of ADV7103 vs. SoC or baseline literature data was consistently demonstrated (per protocol, intention-to-treat, as well as sensitivity analyses). Kalaemia was normalized with ADV7103 with only two doses per day. These analyses were able to show that ADV7103 is superior to the SoC (p<0.0047).

Circassia Pharmaceuticals reported results of a phase III study of Duaklir in chronic obstructive pulmonary disease (COPD), conducted by company partner AstraZeneca. The AMPLIFY study met its co-primary efficacy endpoints with Duaklir demonstrating statistically significant and clinically meaningfully improvements in lung function, measured by forced expiratory volume in one second (FEV1), compared with the combination’s individual component monotherapies (aclidinium and formoterol). The safety profiles of Duaklir and its individual components were consistent with previous studies. Following the completion of the AMPLIFY study, AstraZeneca plans to file a New Drug Application (NDA) for Duaklir with the FDA during the first half of 2018. In addition to AMPLIFY’s positive clinical results, AstraZeneca and Circassia believe the study trial materials’ in vitro comparability data also meet FDA requirements. 

Eli Lilly reported results of a phase III study of galcanezumab for migraine. In the 12-month, open-label study, 270 patients were randomized to treatment with galcanezumab 120mg or galcanezumab 240mg. Galcanezumab was associated with a statistically significant reduction in the number of monthly migraine headache days with both doses (5.6 days for 120mg and 6.5 days for 240mg, p<0.001 for both dosing groups). Notably, there was no clinically meaningful difference in the rate of adverse events between galcanezumab 120mg and 240mg dosing groups. The most commonly reported adverse events (≥10%) in both dosing groups included injection site pain, nasopharyngitis and upper respiratory tract infection. Serious adverse events were reported by three patients in the 120mg dosing group and seven patients in the 240mg group. Lilly plans to submit a Biologics License Application (BLA) to the FDA for galcanezumab in the second half of 2017, followed by submissions to other regulatory agencies around the world.

GTx released results of a phase II, single-arm, open-label trial evaluating enobosarm in postmenopausal women with stress urinary incontinence (SUI). A total of 19 post-menopausal women were enrolled by three clinical site. Enobosarm 3mg (GTx-024) substantially improved stress urinary incontinence (SUI) in women. All 17 patients completing 12 weeks of treatment saw a clinically significant reduction (50% or greater) in stress leaks per day, compared to baseline. Mean stress leaks decreased by 83% from baseline over 12 weeks, and the reductions in daily stress leaks following completion of treatment have been sustained as patients are being followed for up to seven months post-treatment to assess the durability of treatment effect. No patient has relapsed to her baseline levels. The company has initiated a randomized, placebo-controlled phase II clinical trial to evaluate the change in frequency of daily stress urinary incontinence episodes following 12 weeks of treatment. The trial will evaluate the safety and efficacy of enobosarm (1mg and 3mg) compared with placebo in postmenopausal women with SUI. Enobosarm has previously been evaluated in clinical trials enrolling in excess of 1,700 patients, in which approximately 1,200 individuals received doses ranging from 0.1mg to 100mg, and has been observed to be generally safe and well-tolerated.